Efficacy and safety of omalizumab in children and adolescents with moderate-to-severe asthma: A systematic literature review.

BACKGROUND:

There are limited pediatric data about the use of omalizumab, especially the effectiveness and safety ofomalizumab in the real-world management of allergic asthma.

OBJECTIVE:

The objective of this study was to summarize the safety and efficacy of omalizumab in both randomized clinicaltrials (RCT) used for U.S. Food and Drug Administration registration and real-world studies (RWS) based on clinical care of children with moderate-to-severe asthma.

METHODS:

Studies that evaluated omalizumab use in patients < 18 years old and with asthma, published between January2003 and October 2016, were retrieved from medical literature data bases. Assessed outcomes included the following:exacerbation rates, spirometric indices, changes in asthma medication use, asthma control, patient-reported outcomes, andhealth care resource utilization.

RESULTS:

A total of five RWS were identified; outcomes reported were compared with three omalizumab RCTs. Overall, themean rate of annual exacerbations was significantly lower after 6 months to 2 years of treatment with omalizumab in both RCTsand RWS. In two RCTs and three RWS, inhaled corticosteroid use was significantly reduced in patients who used omalizumab.Similar reductions in the use of rescue medication were also observed in the RCTs and RWS on omalizumab. Real-worldevidence demonstrated improvement in forced expiratory volume in the first second of expiration (% predicted) in patientstreated with omalizumab as well as significant improvement in the level of asthma control observed over 1 year. There also wasevidence that omalizumab treatment reduced health care resource utilization, including fewer hospitalizations, emergencydepartment visits, and unscheduled medical visits. Safety outcomes in all five RWS showed no new safety signals anddemonstrated that omalizumab was well tolerated.

CONCLUSION:

Overall, RCT evidence strongly supported omalizumab efficacy and safety as add-on treatment in children 6to 11 years old with moderate-to-severe persistent allergic asthma. RWS data confirmed these findings in an extended patientpopulation of children and adolescents that is more generalizable to the actual day-to-day management of these patients.